Research and Clinical Trials

At Weill Cornell Medicine and NewYork-Presbyterian, we offer a great variety of clinical trials from diagnosis to treatment and beyond. When patients need a transplant, they may have limited options. Clinical trials could offer patients treatments that may save their life.

Weill Cornell Medicine and NewYork-Presbyterian have a Joint Clinical Trials Office (JCTO) to advance clinical research programs. The JCTO provides a coordinated effort to facilitate high impact projects that are synergistic with other institutional goals while ensuring compliance with regulatory standards. The clinical trial program focuses on investigator support and training, study design and feasibility, and facilitating access to resources that streamline clinical trials. The bone marrow and stem cell treatment offers a large number of clinical trials. Many, developed with leading disease experts, focus on treatment of hematological malignancies such as multiple myeloma, lymphoma, leukemia and myeloproliferative disorders, or on sickle cell disease. Other trials address technical aspects of transplantation and aim to make the procedure more accessible, safer and more effective.

The United States Food and Drug Administration (FDA) recently approved the drug ibrutinib for treatment of adults with chronic graft versus host disease (cGVHD). GVHD can occur following a stem cell or bone marrow transplant from a related or unrelated donor, also known as an allogeneic transplant. When the immune cells from the graft (donor) are infused into the body of the host (patient), they may recognize the host’s body tissues as foreign and attack them. Ibrutinib became FDA-approved for treatment of the chronic form of GVHD following clinical trials demonstrating durable safety and effectiveness in patients whose symptoms were resistant to prior corticosteroid treatment administered for immune system suppression.

Defibrotide was studied in multiple clinical trials as a treatment for veno-occlusive disease of the liver (a transplant related liver side effect). Weill Cornell Medicine and NewYork-Presbyterian participated in these studies and physicians co-authored scientific publications. Last year, this drug was approved by the FDA for this particular complication because the clinical trials proved it was life-saving.

Weill Cornell Medicine is participating in clinical trials of CAR-T cells (chimeric antigen receptor T cells), which are revolutionizing the treatment of leukemia as well as lymphoma, and have been recently FDA-approved.

It is the participation of patients in clinical trials that made such progress possible.